Johns Hopkins-Derived Drug Could Transform Heart Failure Treatment

October 31, 2016

InsightOctober 31, 2016

In 2015, two Johns Hopkins researchers and their business partners sold a company they co-founded for $300 million to Bristol-Myers Squibb. The company, Cardioxyl Pharmaceuticals, was formed around an intravenous treatment for late-stage heart failure that has been in the making for 15 years.

It all began when Nazareno Paolocci, a postdoc in cardiology at the time, told his faculty mentor about a compound called HNO, or Angeli’s salt. When Paolocci injected a solution of the salt into dogs, their hearts’ pumping power increased, and their veins and arteries dilated, making it easier for their hearts to work.

Paolocci’s mentor, David Kass, told him the improvement could have been due to any number of things. So Paolocci repeated his experiment and recorded the same positive results. That’s when Kass became a believer.

After publishing papers in 2001 and 2003 on the effects in canines, Paolocci and Kass, along with several pioneers they met in the HNO field, patented the concept in 2004.

Not long after that, Johns Hopkins Technology Ventures connected the group with a venture capitalist and two pharmaceutical company veterans. As a result, Cardioxyl Pharmaceuticals was born in 2005 to develop a compound for human use.

Venture capital flowed in, and a drug went into clinical trials by 2009. After redness appeared on test subjects’ arms at the site of injection, the researchers came up with a new compound. Called CXL-1427, it proved in clinical trials to have a great effect on heart function without any toxicity.

With the trials back on, the group considered its next move. That’s when Cardioxyl was sold to Bristol-Myers Squibb.

If the drug proves effective in further trials, Kass says the ultimate goal would be a treatment for heart failure before it reaches the late stages—something HNO might have the potential to do.